ArticleHuman gene therapy2011
Autologous transplantation of lentivector/acid ceramidase-transduced hematopoietic cells in nonhuman primates.
Article in Human gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 38 citations in OpenAlex.
- A cross-sectional quantitative analysis of the natural history of Farber disease: an ultra-orphan condition with rheumatologic and neurological cardinal disease features.Genetics in medicine : official journal of the American College of Medical Genetics · 2018Pooled it
- Acid Ceramidase Deficiency: Bridging Gaps between Clinical Presentation, Mouse Models, and Future Therapeutic Interventions.Biomolecules · 2023Review
- Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.Molecular therapy. Methods & clinical development · 2022Review
- Hematopoietic stem cell transplant does not prevent neurological deterioration in infants with Farber disease: Case report and literature review.JIMD reports · 2019Article
- Acid ceramidase deficiency: Farber disease and SMA-PME.Orphanet journal of rare diseases · 2018Review
- Deletion of MCP-1 Impedes Pathogenesis of Acid Ceramidase Deficiency.Scientific reports · 2018Article
- Acid Ceramidase Deficiency is characterized by a unique plasma cytokine and ceramide profile that is altered by therapy.Biochimica et biophysica acta. Molecular basis of disease · 2017Article
- Direct Lymph Node Vaccination of Lentivector/Prostate-Specific Antigen is Safe and Generates Tissue-Specific Responses in Rhesus Macaques.Biomedicines · 2016Article
- Preclinical validation: LV/IL-12 transduction of patient leukemia cells for immunotherapy of AML.Molecular therapy. Methods & clinical development · 2016Article
- Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.Human gene therapy · 2014Review
- Odontoid infiltration and spinal compression in Farber Disease: reversal by haematopoietic stem cell transplantation.European journal of pediatrics · 2014Article
- Engineering lentiviral vectors for modulation of dendritic cell apoptotic pathways.Virology journal · 2013Article
- Systemic ceramide accumulation leads to severe and varied pathological consequences.EMBO molecular medicine · 2013Article
- Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes.Gene therapy · 2013Article
- Spinal muscular atrophy associated with progressive myoclonic epilepsy is caused by mutations in ASAH1.American journal of human genetics · 2012Article
- Cell fate control gene therapy based on engineered variants of human deoxycytidine kinase.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
Corrections and comments
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Authors and funding
13 authors at 5 institutions in 4 countries.
Funding
Abstract
Farber disease is a rare lysosomal storage disorder (LSD) that manifests due to acid ceramidase (AC) deficiencies and ceramide accumulation. We present a preclinical gene therapy study for Farber disease employing a lentiviral vector (LV-huAC/huCD25) in three enzymatically normal nonhuman primates. Autologous, mobilized peripheral blood (PB) cells were transduced and infused into fully myelo-ablated recipients with tracking for at least 1 year. Outcomes were assessed by measuring the AC specific activity, ceramide levels, vector persistence/integration, and safety parameters. We observed no hematological, biochemical, radiological, or pathological abnormalities. Hematological recovery occurred by approximately 3 weeks. Vector persistence was observed in PB and bone marrow (BM) cells by qualitative and quantitative PCR. We did not observe any clonal proliferation of PB and BM cells. Importantly, AC-specific activity was detected above normal levels in PB and BM cells analyzed post-transplantation and in spleens and livers at the endpoint of the study. Decreases of ceramide in PB cells as well as in spleen and liver tissues were seen. We expect that this study will provide a roadmap for implementation of clinical gene therapy protocols targeting hematopoietic cells for Farber disease and other LSDs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.